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PTC-775

Chemical Structure : PTC-775

CAS No.:

PTC-775

货号: PC-25280Not For Human Use, Lab Use Only.

PTC-775 is a potent splicing modulator compound (SMC) that correct microtubule-associated protein tau (MAPT) splicing with EC2x value of 4.9 nM in MAPT minigene assay in HEK293T cells (3R MAPT), promotes MAPT exon 10 exclusion, increasing 3R MAPT transcript and protein levels.

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纯度 & COA & 质检文件 纯度: >98% (HPLC)

生物&药学活性

PTC-775 is a potent splicing modulator compound (SMC) that correct microtubule-associated protein tau (MAPT) splicing with EC2x value of 4.9 nM in MAPT minigene assay in HEK293T cells (3R MAPT), promotes MAPT exon 10 exclusion, increasing 3R MAPT transcript and protein levels.
PTC-775 induces a dose-dependent increase in 3R tau in MSD protein assay EC1.3X value of 8.9 nM.
PTC-775 lowers 4R MAPT expression and rescues tauopathy phenotypes in the S305N iPSC-neuronal model.
PTC-775 also significantly protected neurons against stress conditions.

物理化学性质&存储条件

分子量
分子式
外观性状 Solid
CAS No.
储存条件
固体粉末
-20°C 12 个月; 4°C 6 个月
配置液
-80°C 6 个月; -20°C 6 个月
Shipping
Solubility

10 mM in DMSO

Chemical Name/SMILES

参考文献

1. Silva MC, et al. bioRxiv [Preprint]. 2025 May 10:2025.05.06.652428.

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